ESPE2024 Poster Category 3 Growth and Syndromes (34 abstracts)
Efficacy of alternative Somatropine-rhGH treatment for the management of SHOX-related short stature
Laura Guazzarotti 1 , Maria Felicia Faienza 2 , Francesco Gallo 3 , Rossella Gaudino 4 , Maria Cristina Maggio 5 , Gabriella Pozzobon 6 , Mariacarolina Salerno 7 , Malgorzata Wasniewska 8 & Marco Cappa 9
1Head of Pediatric Endocrinology Unit, Women's and Children's Health Department University Hospital of Padua, Padua, Italy. 2Pediatric Unit, Department of Precision and Regenerative Medicine and Ionian Area, University of Bari "Aldo Moro", Bari, Italy. 3Pediatric Unit, Antonio Perrino Hospital, Brindisi, Italy. 4Pediatric Unit, Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, University of Verona, Verona, Italy. 5PROMISE Department, University of Palermo, U.O.S. of Pediatric Rheumatology, Pediatric Clinic – General Paediatrics, “G. Children's Hospital”. Di Cristina”, ARNAS, Palermo, Italy. 6Pediatric Unit - San Raffaele Hospital Milan Vita - Salute San Raffaele University, Milan, Italy. 7Pediatric Endocrinology Unit, Department of Translational Medical Sciences- University of Naples Federico II, Naples, Italy. 8Department of Human Pathology of Adulthood and Childhood, University of Messina, Italy. A.O.U. Policlinico "G. Martino", Messina, Italy. 9Head of Research Area for Innovative Therapies in Endocrinopathies Bambino Gesù Children's Hospital, IRCCS, Rome, Italy
Introduction: The short stature homeobox-containing gene, SHOX, encodes a homeodomain transcription factor responsible for a significant proportion of long-bone growth. Growth impairment is a common manifestation in SHOX deficient patients (SHOXD), with variable degrees of height disablement. The growth promoter treatment of SHOXD patients consists in the administration of recombinant human GH (rhGH). In Italy, the allowed formulation of rhGH (somatropine, Humatrope®) was unavailable for a long period. Accordingly, Italian Health authorities (AIFA) allowed clinicians to use an alternative rhGH.
Objective: Our objective was to evaluate growth rate during an alternative rhGH treatment compared to the growth rate obtained with Humatrope®.
Methods: Forty-eight SHOXD patients (24 female) aged 2 – 17 years treated for short stature with rhGH Humatrope® were switched to an alternative somatropine rhGH treatment. Mean of height standard deviation score (SDS) and mean of height velocity SDS were recorded at 12 and 6 months before and after the switch to evaluate the differences in the growth rate.
Results: Results from this preliminary analysis showed that mean of height velocity growth SDS scores were 0.06 (standard deviation, SD, 2.37), 0.49 (SD 1.99) in the 12 and 6 months before the switch, respectively; 0.47 (SD 2.35) at the moment of the switch and 0.36 (SD 2.6), 0.05 (SD 1.83) in the 6 and 12 months after the switch, respectively. Mean of height growth SDS scores were -1.69 (SD, 0.80), -1.43 (SD 1.10) in the 12 and 6 months before the switch, respectively; -1.46 (SD 0.85) at the moment of the switch and -1.22 (SD 0.87), -1.34 (SD 0.84) in the 12 and 6 months after the switch, respectively. The growth data analyzed did not show a significant statistical difference after 6 and 12 months of brand rhGH switch.
Conclusion: In our experience, the switch from Humatrope® to the alternative rhGH somatropine resulted in a similar response to treatment regarding both height velocity growth and height growth. This may provide evidence for an alternative rhGH brand to use in patients affected by SHOX-related short stature. Further analysis on safety and compliance aspects related to the alternative rhGH treatment will be evaluated.
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